Amyotrophic lateral sclerosis (ALS) is a degenerative motor neuron disorder that leads to progressive muscle wasting and weakness. The typical survival time is three to five years from onset. The FDA’s 2017 approval of Mitsubishi Tanabe’s Radicava resulted in the second approved treatment option for ALS, yet pressing unmet need remains in this arena. The ALS pipeline comprises diverse programs designed to be neuroprotective, to enhance respiratory function, or to restore lost neurons in the spinal cord; however, clinical success in ALS has historically been a formidable challenge. Nonetheless, clinical and commercial opportunity exists for developers that can overcome the scientific and clinical hurdles blocking the path to regulatory approval for the treatment of this devastating disease.

QUESTIONS ANSWERED

  • How has Mitsubishi Tanabe’s Radicava been incorporated into the treatment of U.S. ALS patients? What are the key advantages and disadvantages of the product?
  • How will the size of the drug-treated ALS population change through 2028?
  • What are the key unmet treatment needs in ALS?
  • Which emerging therapies have ALS experts most intrigued or optimistic? How would new therapies influence management of ALS patients?
  • How are emerging drugs being evaluated, and which are likely to launch by 2028? What commercial impact will they have on the ALS market?

PRODUCT DESCRIPTION

Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.

Scope:

  • Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom.
  • Primary research: Six country-specific interviews with ALS neurologists.
  • Epidemiology: Diagnosed prevalence, diagnosed prevalence by disease type (familial or sporadic ALS), diagnosed prevalence by comorbid frontotemporal dementia.
  • Emerging therapies: Phase III/Registered: 5; Phase II & I/II: 6; coverage of select preclinical and Phase I products.
  • Market forecast and alternative market scenarios: Drug-level sales and patient share of ALS therapies in 2028.
  • Key companies: Mitsubishi Tanabe Pharma, Biogen, Ionis, Orphazyme, Orion Pharma, AB Science, Neuralstem, BrainStorm Therapeutics.
  • Key drugs: Radicava (edaravone), BIIB-067, arimoclomol, levosimendan, masitinib, riluzole, NSI-566, mesenchymal stem cells expanded and induced to secrete neurotrophic factors (MSC-NTF cells).

Table of contents

  • Disease Landscape & Forecast
    • COVID-19
    • Executive Summary
      • Key Findings
    • Commercial Outlook
      • Key Findings
      • Drivers and Constraints
        • What Factors Are Driving Sales in Amyotrophic Lateral Sclerosis?
        • What Factors Are Constraining Sales in Amyotrophic Lateral Sclerosis?
      • Drug-Class-Specific Trends
        • Free Radical Scavenger
        • Antisense Oligonucleotide
        • Stem Cell Therapy
        • Molecular Chaperone Activators
    • Forecast
    • Etiology and Pathophysiology
      • Etiology and Pathophysiology
        • Disease Overview
        • Expert Insight
      • Etiology
        • Etiology of Amyotrophic Lateral Sclerosis
        • Select Genes Associated with Amyotrophic Lateral Sclerosis
      • Pathophysiology
        • Pathophysiology of Amyotrophic Lateral Sclerosis
      • Disease Presentation
      • Overlap of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
      • Disease Burden
      • Prognostic Factors
      • Key Pathways and Drug Targets
    • Epidemiology
      • Introduction
        • Key Findings
      • Epidemiology Populations
        • Diagnosed Prevalent Cases of Amyotrophic Lateral Sclerosis
        • Diagnosed Prevalent Cases of Amyotrophic Lateral Sclerosis by Subtype
        • Drug-Treated Prevalent Cases of Amyotrophic Lateral Sclerosis
    • Current Treatment
      • Key Findings
      • Diagnosis
        • Overview
        • Treatment Providers and Referral Patterns for Amyotrophic Lateral Sclerosis
      • Treatment Goals
        • Key Endpoints Used in Clinical Trials for Amyotrophic Lateral Sclerosis
      • Key Current Therapies
        • Riluzole
        • Nuedexta
        • Edaravone
        • Key Symptomatic Therapies for Amyotrophic Lateral Sclerosis
      • Medical Practice
        • Overview
        • Treatment Guidelines
        • Nonpharmacological Interventions for Amyotrophic Lateral Sclerosis
        • Multidisciplinary Care
        • Region-Specific Treatment
    • Unmet Need Overview
      • Key Findings
        • Expert Insight
      • Attainment of Unmet Needs
        • Current Attainment of Unmet Needs in Amyotrophic Lateral Sclerosis
        • Future Attainment of Unmet Needs in Amyotrophic Lateral Sclerosis
    • Emerging Therapies
      • Key Findings
        • Pipeline Overview
      • Key Emerging Therapies
        • Stem Cell Therapy
        • Antisense Oligonucleotide
        • Molecular Chaperone Activators
        • Tyrosine Kinase Inhibitors
        • Combination Therapy for Amyotrophic Lateral Sclerosis
        • Troponin Stimulants
        • Calcium-Binding Protein Modulators
      • Early-Phase Pipeline Analysis
      • Key Discontinuations and Failures in Amyotrophic Lateral Sclerosis
      • Patient Registries
      • Orphan-Drug Designation
        • Orphan Drug Provisions: United States
        • Orphan Drug Provisions: Europe
    • Access and Reimbursement Overview
      • Reimbursement of Current Amyotrophic Lateral Sclerosis Therapies
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
    • Methodology
      • Bottom-Up Forecasting Overview
        • Bottom-Up Market Forecasting Overview
        • Patient Share and Sales Calculations
        • Drug-Specific Assumptions
      • Bottom-Up Forecast Assumptions
        • General Sources of Data
        • Drug-Treatable Rate Assumptions in Amyotrophic Lateral Sclerosis
        • General Statements About Pricing
        • Dosing, Days of Therapy, and Compliance
        • Generic Erosion
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Amyotrophic Lateral Sclerosis Bibliography

Author(s): Natasha Bardhan, M.Pharm; Swarali Tadwalkar, MPH

Natasha Bardhan is a Research Associate for the Infectious, Niche, & Rare Diseases (INRD) team at Decision Resources. Natasha Bardhan holds a bachelor’s degree in Pharmacy from DIPSAR, New Delhi and has completed her master’s in Pharmaceutics from Amity University, Noida. Prior to joining DRG, she worked as a Senior Associate at WNS (Healthcare) for two and a half years. She has worked on multiple client projects involving patents, sales and forecasting, pipeline analysis, keywords research and product/company profiling.

Swarali joined Decision Resources Group (DRG) in 2016 and with the Epidemiology team develops epidemiological populations forecasts for different infectious and non-communicable diseases with her particular interests in the oncology space. Prior to joining DRG, she has been extensively involved in primary and secondary healthcare research. Her experience involves projects in digital health, health policy and management, and health economics and outcomes research (HEOR). She has also coordinated various non-governmental public health projects focusing in hepatitis and human papilloma virus treatment access. Swarali holds a Masters in Public Health (Epidemiology) degree from the University of South Florida, Tampa.